Publications by authors named "Angelique S Camp"
Article Synopsis
- Efficient gene transfer is crucial for treating Duchenne muscular dystrophy (DMD), and the first clinical trial used a new chimeric adeno-associated virus (AAV) capsid variant called AAV2.5, which enhances muscle targeting while minimizing immune response.
- This phase I trial involved injecting AAV2.5 into the bicep of boys with DMD and comparing results to saline control injections, with some patients receiving an empty AAV capsid to differentiate immune responses.
- Results showed that AAV2.5 was safe, well tolerated, and effectively delivered genes, paving the way for customized AAV vectors to improve gene therapy approaches for DMD and potentially other conditions.
